International coverage of treatments and trials, with the ability to zoom in on countries we cover. From approved options to compassionate access.
The weekly Breakthrough Brief on treatments and trials. Every email has an unsubscribe link.
Series
Four access-first episodes on acne in 2026: what is labelled, where it is labelled, and how someone 12 or older actually gets it. International by default. Zoom by country. Authorized is not funded.
Hyrnuo (sevabertinib) for HER2 TKD-mutant NSCLC
The FDA and Health Canada have labelled an oral HER2 tyrosine-kinase inhibitor for adults with locally advanced or metastatic NSCLC whose tumours have HER2…
The FDA and Health Canada have labelled an oral HER2 tyrosine-kinase inhibitor for adults with locally…
This is for families of children (and adults) with genetically confirmed AADC deficiency from biallelic DDC changes who have achieved skull maturity and may be candidates for labelled US Kebilidi…
This is for people living with recessive dystrophic epidermolysis bullosa — and the caregivers who help them — who have chronic open wounds and may be candidates for labelled US Zevaskyn autologous…
This is for adults living with recurrent respiratory papillomatosis who still need repeated airway surgeries and may be candidates for labelled US Papzimeos — four under-the-skin shots over about 12…
This is for families of children (and adults) with genetically confirmed Wiskott-Aldrich syndrome who need a transplant-style path and do not have a suitable HLA-matched related donor — and who…
This is for families of children with Sanfilippo syndrome type A (MPS IIIA) who still have preserved neurodevelopmental function and may be candidates for labelled US Fayuvi one-time IV gene therapy…
This is for adults with type 1 diabetes who already have chronic kidney disease with protein in the urine and may be candidates for labelled US Kerendia as a once-daily oral add-on on top of ACE…
This is for caregivers of children 4–11 years with genetically confirmed APDS who weigh at least 27 kg (about 60 lb) and who may be candidates for labelled US Joenja as a twice-daily oral…
This is for children and adults living with SMA who are already on an SMN2-targeted treatment (such as nusinersen or risdiplam) and who may be candidates for labelled US Isembyld as a…
This is for adults with type 2 diabetes who are at high risk for heart attack, stroke, or cardiovascular death, and who may already be candidates for weekly Mounjaro for blood sugar — now with a…
The weekly Breakthrough Brief on treatments and trials. Every email has an unsubscribe link.